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Vertex cystic fibrosis drug wins US FDA approval for use in children aged 2 to 5

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Vertex cystic fibrosis drug wins US FDA approval for use in children aged 2 to 5

Vertex Pharmaceuticals Inc. said the U.S. Food and Drug Administration approved its medicine Orkambi to treat certain children with the most common form of cystic fibrosis.

The Boston-based company said in an Aug. 7 press release that Orkambi is now approved for children aged 2 to 5 years with cystic fibrosis who have two copies of the F508del-CFTR gene mutation, making it the first medicine approved for treating the underlying cause of the disease in this population.

Cystic fibrosis is a rare, life-threatening genetic condition that causes mucus buildup in the lungs, pancreas and other organs. The disease affects about 75,000 people in North America, Europe and Australia.

Vertex said the FDA approval was based on data from a phase 3 trial in 60 patients, which showed that treatment from Orkambi was generally safe and well tolerated for 24 weeks, with a safety profile similar to that in patients ages 6 years and older.

The company added that it has submitted a marketing authorization application to the European Medicines Agency to extend the use of Orkambi in children ages 2 through 5 years, with a decision expected in the first half of 2019.

Orkambi, which generated revenues of about $311 million in the second quarter of 2018, is already approved in the U.S. for treating cystic fibrosis in patients ages 6 and older who have two copies of the F508del-CFTR gene mutation.